The FDA has approved Fayuvi as the first gene therapy for children with Sanfilippo syndrome type A, introducing the first treatment designed to address the underlying cause of the rare neurodegenerative disorder.

Sudarshan Pharma has approved the acquisition of a 9.5% stake in US-based MedTherapy Biotechnology, expanding its exposure to oncology cell and gene therapy.

The commercial partnership combines 4basebio’s cell-free hpDNA platform with Genezen’s viral vector CDMO capabilities to provide cell and gene therapy developers with an alternative to conventional plasmid DNA manufacturing.

The approval introduces the first therapy designed to address the underlying cause of glycogen storage disease type Ia, offering a one-time gene therapy option for eligible patients aged eight years and older.

The acquisition would add a BLA-stage, one-time AAV gene therapy to PTC’s rare disease portfolio, with a rolling US submission expected to complete in the fourth quarter of 2026.